- Graft-vs-Host Disease (GvHD) is a severe complication of allogeneic hematopoietic stem cell transplantation (HSCT) that occurs when the donated (graft) cells recognize the host’s tissues and organs as foreign and attack them.
- Every year around 25.000 allogeneic HSCTs are performed worldwide as a treatment for hematologic cancers, bone marrow disorders and immunodeficiencies.
- Around half of HSCT patients will develop acute form of GvHD (aGvHD). Further 50% of HSCT patients will develop chronic form of GvHD (cGvHD) with or without preexisting aGVHD.
- Steroids are the fist line of treatment, nevertheless more than 50% of patients are resistant to steroid treatment. There are no approved second line treatment options. Treatment rely mostly on off-label drugs that have little or no efficacy and often cause severe side effects.
- If left untreated, GvHD will often lead to multiple organ failure and death.
Due to its systemic immunomodulatory / immunosuppressive effect, apceth™-201 has a promising potential as a new therapy for GvHD with a low toxicity and potentially no side effects.
- Glioblastoma multiforme (GBM, also known as Grade IV astrocytoma) is the most prevalent and aggressive brain tumor, representing up to 40% of primary brain tumors.
- GBM is an orphan disease with an annual incidence of 2-3 cases per 100.000 people in Europe and the US. Still, due to its extremely aggressive nature and lack of effective treatments, GBM is a disease with very few therapeutic options and high medical need.
- Even though GBM incidence increases with age, the disease can be diagnosed at any age, including early childhood.
- Current standard of care of newly diagnosed patients includes surgery, radiotherapy and classical chemotherapy that offers only incremental improvements in the patient health.
- The overall prognosis is poor; almost all GBM patients relapse and progress, even with the best standard of care:
– The median survival time is approx. 10 to 14 months.
– Only a third of patients survive 1 year following diagnosis, and less than 5% live beyond 5 years.
– Patients with recurrent GBM have a median survival time of 5 to 7 months.
apceth™-301 therapy is a potent local activator of the immune system. The preclinical data show capability of apceth™-301 not only to stimulate immune response to eradicate GBM tissue, but also to induce anti-tumor immunity in animal models with GBM.
- Inflammatory bowel disease (IBD) is a group of autoimmune disorders causing chronic inflammation of the digestive system.
- The two most common types of IBD are Crohn’s disease and ulcerative colitis (UC). While Crohn’s disease effects only large intestine, UC can effect any part of the digestive system.
- Up to 3 million individuals suffer from IBD in Europe.
- The majority of IBD diagnosis are made in adolescence and early adulthood.
- The disease can range from mild to severe. It can significantly reduce quality of life of affected individuals and may increase incidence of bowel cancer. In severe cases surgery to remove inflamed and damaged segments of the digestive system is necessary.
apceth™-201 shows promising therapeutic effect in preclinical animal models of IBD, which is based on apceth™-201 ability to modulate the immune system, modulating the underlying autoimmune (inflammatory) process.
- Chronic Obstructive Pulmonary Disease (COPD) is a lung disease characterized by lung inflammation, which leads to progressive lung damage and limitations in lung airflow.
- The main symptoms include chronic cough, wheezing, shortness of breath, chest discomfort and fatigue.
- The primary cause of COPD is smoking or exposure to tobacco smoke, but also to indoor and outdoor pollution, occupational dusts and chemicals, or recurrent lower respiratory infections particularly during childhood.
- WHO estimates that 65 million people globally have moderate to severe COPD, that will become the third leading cause of death worldwide by 2030.
- There is currently no cure for COPD and no treatment that will stop its progression.
Due to its immunomodulatory effect, apceth™-201 therapy shows a promising potential in mitigating the chronic inflammation process, as well as in improving the lung function in preclinical animal models of COPD.
- Type 1 diabetes is an autoimmune disease that results in the destruction of insulin-producing beta-cells in the pancreas. Insulin is an essential hormone that mediates the transport of glucose from the blood into the cells. Absence of insulin results in elevated levels of sugar in the blood, ketoacidosis, and death unless treated by insulin injections.
- Even if properly treated with insulin, patients will develop long-term complications such as increased incidence of cardiovascular disease, stroke, chronic kidney disease, foot ulcers resulting in a reduced quality of life and shorter life expectancy.
- Type 1 diabetes can occur at any age, but most often it is diagnosed during childhood and adolescence. Up to 10% of diabetic patients are type 1.
- According to the WHO, the number of type 1 diabetic individuals has risen from 108 million in 1980 to 422 million in 2014 while the prevalence has risen from 4.7% in 1980 to 8.5% in 2014.
apceth™-201 shows promising effect in the prevention of disease onset and preservation of beta-cell function in type 1 diabetes animal models.